NEJM: Study on differences in the therapeutic dose of hydroxylurea in sickle cell anemia

Hydroxyurea has been shown to be highly safe, feasible and effective in children'ssickle cell anemia in sub-Saharan Africa, reducing the incidence and mortality ofvascularocclusionHowever, the dose standard for hydroxyurea therapy has not yet been determined, and the clinical effects of increased doses are not clearrecently researchers compared 20 mg/kg of body weight daily (fixed dose group) or up to 30 mg/kg of body weight daily (elevated dose group), the clinical difference between the two dosesThe main endpoint of the study was 24 months after hemoglobin levels of 9.0 g or more per litre or 20% or more of fetal hemoglobinSecondary endpoints are malaria, vascular occlusion events, and severe adverse events94 children received a fixed dose (average age 4.6 years), 93 received a high dose (average age 4.8 years), and the average dose of hydroxyrewas was 19.2 mg/kg and 29.5 mg/kgAt the end of the trial, 86% of the children in the elevated dose group met the dose threshold, while only 37% of the children in the fixed dose group met the dose thresholdThe incidence of sickle cell-related adverse events (0.43), vascular occlusion pain (0.43), cases of acute chest syndrome or pneumonia (0.27), infusion (0.30) and hospitalization (0.21) were all low in children in the elevated dose groupThe dose-related toxicity events identified in the inter-group laboratory were similar and there were no serious neutrophil reduction or platelet reduction eventsstudies have found that for children with sickle cell anemia in sub-Saharan Africa, the clinical effect of elevated dose hydroxyurea treatment is better than that of fixed-dose programmes